USA Food and Drug Agency (FDA) approved Trikafta (elexacaftor / ivacaftor / tezacaftor), a first triple-combination therapy, which will be available for treatment of patients with the most common mutation in cystic fibrosis (CF).
Trikafta was approved for patients at 12 years of age, or more, who have at least one copy of F508del mutation in cystic fibrosis transmembrane conductance regulator (CFTR)gene, which corresponds to 90% of population with CF.
As stated by Norman Sharpless, FDA acting commissioner – “This decision allows for a new method for treatment of patients, including adolescents, and provides access to additional effective therapy”. He also added, that in the recent years, they have been witnessing a significant progress in treatment of CF, and an improvement in quality of life of patients, however many patient groups did not have approved treatment options. This was the reason behind using all available programs, to maximize the rate for approval of the treatment, while maintaining high treatment standards.
The effectiveness of Trikafta in patients with CF at 12 years of age or older, was demonstrated in two studies. The first study was a 24-week randomized double-blinded placebo-controlled trial, with 403 patients with, F508del mutation. Second study was a 4-week randomized double-blinded study with an active control, in 107 patients, with two identical F508delmutations.
The information for indication of Trikafta includes warnings, associated with risks of abnormal liver function (transaminases, and bilirubin), parallel use with other products, which act as inducers or inhibitors of another hepatic enzyme, cytochrome CYP3A, and cataract development.
Patients and healthcare personnel must discuss all details of treatment, prior to its administration.
Trikafta approval was handled by Vertex Pharmaceuticals Incorporated, which will also be receiving and additional voucher for acceleration of therapy development.